RARE Daily

Bayer Strengthens Gene Therapy Portfolio with Access to Acuitas LNP Technology

June 8, 2023

Rare Daily Staff

Bayer has entered into a development and license agreement option with Acuitas Therapeutics to use the company’s lipid nanoparticle delivery systems to support Bayer’s in vivo gene editing and protein replacement programs with the goal of delivering RNA payloads to the liver.

Financial details were not disclosed.

Liquid nanoparticles (LNPs) are spheric drug delivery bodies that can be equipped with therapeutic payloads for intracellular delivery. Acuitas’ proprietary LNP technology is used in multiple vaccines and therapeutics in clinical development and was also used in some COVID-19 vaccines that were approved and administered to people in 180 countries.

The delivery technology protects the mRNA payload after administration allowing it to be safely and effectively delivered into cells. In addition to mRNA, Acuitas’ LNPs can be used to deliver a range of different nucleic acid therapeutics including small interfering RNA (siRNA), antisense oligonucleotides and DNA.

Through the development and option for license agreement, Bayer and its gene therapy focused affiliate Asklepios BioPharmaceutical (AskBio) will gain access to Acuitas’ high potency ionizable lipid technology and LNP carriers to develop efficient, targeted, and transient delivery of gene editing RNA components to the liver.

Gene editing is the targeted manipulation of genetic material. It enables a range of edits to the DNA allowing a diverse range of therapeutic applications. Gene editing can be applied inside and outside the patient to treat a variety of diseases and provide diverse clinical benefits.

Gene editing as therapeutic treatment of genetic diseases can be used ex vivo to treat genetic dysfunctions, where a patient’s cells are removed from the body, modified, and re-administered, or in vivo, where modifications are made directly within the body. To deliver the different components needed for in vivo gene editing to the right place in the patient’s body, transport vehicles, such as LNPs can be used.

“Complementing in-house expertise with external collaboration continues to be a priority in areas of high unmet medical need where insufficient, or no treatment options are currently available,” said Friedemann Janus, acting head of business development and licensing/open innovation for the Pharmaceuticals Division of Bayer. “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

Photo: Friedemann Janus, acting head of business development and licensing/open innovation for the Pharmaceuticals Division of Bayer

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